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在接受基于venetoclax的非密集治疗的NPM1-突变AML患者中,分子MRD的预后强烈

Jad Othman^1,2,3, Ing S Tiong^4,5,6, Jenny O'Nions⁷

¹Department of Medical and Molecular Genetics, King's College London, London, United Kingdom.

|August 30, 2023

概括

可测量的残留疾病 (MRD) 监测对于接受venetoclax治疗的NPM1-突变急性髓性白血病 (AML) 患者至关重要. 到第4周期达到MRD阴性,显著改善整体存活率,突出其预后价值.

科学领域:

血液学血液学血液学
在瘤学瘤学.
分子诊断学分子诊断

背景情况:

对可测量的残留疾病 (MRD) 的定量逆转录聚合酶连锁反应 (qRT-PCR) 是NPM1-突变性急性髓性白血病 (AML) 预后治疗的强化化疗.
缺乏基于venetoclax的非密集治疗NPM1-突变AML的MRD实用性的数据,尽管该疗法在这种基因型中具有很高的疗效.

研究的目的:

分析NPM1 MRD在以前未经治疗的NPM1-突变AML患者接受venetoclax组合疗法的现实世界队列中的预后影响.
评估MRD阴性和临床结果之间的关联,包括整体存活率和无治疗缓解.

主要方法:

对76名先前未经治疗的NPM1-突变AML患者的国际真实世界队列的分析.
患者通过使用venetoclax和低甲基化剂 (HMAs) 或低剂量cytarabine (LDAC) 实现完全缓解 (CR) 或CR与不完全的血液恢复 (CRi).
在多个时间点使用qRT-PCR评估了骨髓 (BM) 的MRD水平.

主要成果:

58%的患者获得了BM MRD负面反应,18%的患者从基线获得了≥4 log10的减少.
在第6周期结束时,BM MRD累积负面率达到50%.
在第4个周期结束时,获得BM MRD阴性患者的2年整体存活率为84%,而MRD阳性患者的这一比例为46%.
在多变量分析中,MRD阴性是最强的预后因素.
22名患者在MRD阴性缓解中选择性停止治疗,达到88%的2年无治疗缓解率.

结论:

评估NPM1MRD为NPM1-突变AML患者通过venetoclax组合疗法实现缓解提供了有价值的预后信息.
达到MRD阴性,特别是到第4周期,与显著改善的整体存活率有关.
在大量获得MRD阴性患者中,可以实现无治疗缓解.

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